ArticleBlood2014
Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells.
Article in Blood, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 56 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
56 citing papers in PubMed, 83 citations in OpenAlex.
- Combined effects of restriction factors and transduction adjuvants on lentiviral vector gene transfer efficacy.Scientific reports · 2025Article
- Expanding the frontier of CAR therapy: comparative insights into CAR-T, CAR-NK, CAR-M, and CAR-DC approaches.Annals of hematology · 2025Review
- Tracking HIV persistence across T cell lineages during early ART-treated HIV-1-infection using a reservoir-marking humanized mouse model.Nature communications · 2025Article
- Advancements in Hematopoietic Stem Cell Gene Therapy: A Journey of Progress for Viral Transduction.Cells · 2024Review
- Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Lentiviral-Mediated Systemic RNA Interference In Vivo.Methods in molecular biology (Clifton, N.J.) · 2024Article
- Identification of a small molecule for enhancing lentiviral transduction of T cells.Molecular therapy. Methods & clinical development · 2023Article
- CAR Gene Delivery by T-cell Targeted Lentiviral Vectors is Enhanced by Rapamycin Induced Reduction of Antiviral Mechanisms.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2023Article
- A straightforward microfluidic-based approach toward optimizing transduction efficiency of HIV-1-derived lentiviral vectors in BCP-ALL cells.Biotechnology reports (Amsterdam, Netherlands) · 2023Article
- Interferon-inducible phospholipids govern IFITM3-dependent endosomal antiviral immunity.The EMBO journal · 2023Article
- Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.Seminars in immunology · 2023Review
- Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells.Frontiers in genome editing · 2023Review
- Chimeric antigen receptor natural killer (CAR-NK) cell design and engineering for cancer therapy.Journal of hematology & oncology · 2021Review
- Gene therapy using haematopoietic stem and progenitor cells.Nature reviews. Genetics · 2021Review
- Cardiac Aging: From Basic Research to Therapeutics.Oxidative medicine and cellular longevity · 2021Review
- CAR-NK Cells: From Natural Basis to Design for Kill.Frontiers in immunology · 2021Review
- Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy.Gene therapy · 2020Article
- Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice.Blood advances · 2020Article
- Review
- Enhanced Generation of Induced Cardiomyocytes Using a Small-Molecule Cocktail to Overcome Barriers to Cardiac Cellular Reprogramming.Journal of the American Heart Association · 2020Article
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Authors and funding
13 authors at 3 institutions in 1 country.
Funding
Abstract
Transplantation of genetically modified hematopoietic stem cells (HSCs) is a promising therapeutic strategy for genetic diseases, HIV, and cancer. However, a barrier for clinical HSC gene therapy is the limited efficiency of gene delivery via lentiviral vectors (LVs) into HSCs. We show here that rapamycin, an allosteric inhibitor of the mammalian target of rapamycin complexes, facilitates highly efficient lentiviral transduction of mouse and human HSCs and dramatically enhances marking frequency in long-term engrafting cells in mice. Mechanistically, rapamycin enhanced postbinding endocytic events, leading to increased levels of LV cytoplasmic entry, reverse transcription, and genomic integration. Despite increasing LV copy number, rapamycin did not significantly alter LV integration site profile or chromosomal distribution in mouse HSCs. Rapamycin also enhanced in situ transduction of mouse HSCs via direct intraosseous infusion. Collectively, rapamycin strongly augments LV transduction of HSCs in vitro and in vivo and may prove useful for therapeutic gene delivery.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.